ROLE OF REGULATORY TYPE 1 CELLS IN IMMUNE TOLERANCE

An important adverse effect that limits the success of gene therapy is the immune response towards genetically modified cells. This response is mainly mediated by a specific subset of cells known as T lymphocytes. Several strategies have been explored to limit the anti-transgene immune response, but none of them is able to provide long-term protection of genetically modified cells. A novel subset of T cell, known as regulatory T (Tr) cells, represents a promising candidate for the development of novel approaches to suppress immune responses and to prevent the clearance of genetically modified cells. The aim of this proposal is to further investigate the mechanisms by which Tr cells can be induced in vitro and to characterize them at both the molecular and the cellular level. Ultimately, this work will contribute to improve our knowledge of the biology of regulatory T cells and to develop strategies to generate transgene-specific regulatory T cells to be used as “cellular therapy” to prevent clearance of corrected cells after gene therapy.